Editas Medicine Q1 2023 Earnings Call Transcript

Quartr
Provided by Quartr
May 5, 2023

There are 16 speakers on the call.

Operator

Good morning, and welcome to Editas Medicine's First Quarter Conference Call. All participants are now in a listen only mode. I would now like to turn the call over to Kristie Barnett, Corporate Communications and Investor Relations at Editas Medicine.

Speaker 1

Thank you, Camilla. Good morning, everyone, and welcome to

Speaker 2

our Q1 2023 conference call.

Speaker 1

Earlier this morning, we issued a press release providing our financial results and recent corporate updates. A replay of today's call will be available in the Investors section of our website approximately 2 hours after its completion. After our prepared remarks, we will open the call for Q and A. As a reminder, various remarks that we make during this call about the company's future expectations, plans and prospects constitute forward looking statements for purposes of the Safe Harbor provisions under the Private Securities Litigation Reform Act of 1995. Actual results may differ materially from those indicated by these forward looking statements as a result of various important factors, including those discussed In the Risk Factors section of our most recent annual report on Form 10 ks, which is on file with the SEC as updated by our subsequent filings.

Speaker 1

In addition, any forward looking statements represent our views only as of today and should not be relied upon as representing our views as of any subsequent date. Except as required by law, we specifically disclaim any obligation to update or revise Any forward looking statements, even if our views change. Now, I will turn the call over to our CEO, Gilmore O'Neill.

Speaker 3

Thank you, Christy, and good morning, everyone. Thank you for joining us today on Editas' Q1 earnings call. I'm joined today by 2 other members of the Editas executive team, Baisong Mai, our Chief Medical Officer and Michelle Robertson, our Chief Financial Officer. As many of you know, in early January, we shared our strategy to position Editas as a leader in in vivo programmable gene editing and hemoglobinopathies. During the Q1, we successfully executed this strategy, driving to our goal of delivering life changing medicines patients with previously untreatable or undertreated diseases.

Speaker 3

We are increasing our momentum in driving our ex vivo EDIT-three zero one program As we pursue a leadership position in hematopoietic stem cell medicines for hemoglobinopathy. As a quick recap, there are 3 underlying pillars To our new strategy, first, while continuing to develop EDIT-three zero one for severe sickle cell disease and transfusion dependent beta thalassemia or TDT, We have sharpened our discovery focus to in vivo administered genome editing medicines. As part of that refocusing effort, We previously announced that we had divested our INK Cell franchise to Shoreline Biosciences in January. 2nd, We are strengthening our discovery engine and technological capabilities. We have divided our research division into separate technology and drug discovery groups, enhancing the capabilities of each and implementing our new target selection criteria.

Speaker 3

Finally, our 3rd strategic pillar is an increase and expanded approach to business development. In tandem, we will continue to deleverage our IP portfolio to drive out licensing and partnership discussions. So How have we executed against our new strategy in the Q1? We have increased our investment in our EDIT-three zero one program after reviewing promising initial RUBY Phase III study data that indicated that we have a competitive and potentially differentiated program to treat sickle cell anemia and TDT. Additionally, We are investing to develop an in vivo approach for editing hematopoietic stem cells for the treatment of sickle cell disease and TDT, leveraging the unique And differentiated approach of EDIT-three zero one that we have already seen POC for in humans.

Speaker 3

We continue to ramp up enrollment and dosing of patients in the RUBY trial for sickle cell disease and are on track to have dosed 20 total patients by the end of 2023. We are also excited to share that the FDA recently granted orphan drug designation to EDIT-three zero one for the treatment of sickle cell disease And we are pleased to announce that in June, we will provide a Rubi clinical data update in an oral presentation at the European Hematology Association or EHA Congress and in our company sponsored webinar. Baisong will share further details regarding our June data readout and our enrollment progress in his remarks. On EDIT-three zero one for TDT, we are pleased to share that we dosed the first patient in our Edithyle Phase onetwo trial in the Q1 and that the patient has Successfully engrafted neutrophils and platelets. Enrollment continues to progress and we remain on track to provide initial clinical data from the Edithyle trial year end.

Speaker 3

Moving to in vivo, earlier this year, our drug discovery group began lead discovery work on in vivo therapeutic targets in HSCs For hematopoietic stem cells and other tissues. As a reminder, under our new target selection criteria, we will select therapeutic targets that will allow Our genome editing approach to differentiate maximally from the current standard of care for serious diseases. The target selection criteria will work to ensure targets Are selected that maximize the probability of technical, regulatory and commercial success. Our search for a new CSO to lead this drug discovery group continues to progress, I look forward to updating you on this search and our in vivo work in the future. Turning to our intellectual property position.

Speaker 3

Since the founding of Editas, we have placed substantial importance in securing robust intellectual property protections covering our cutting edge scientific discoveries and gene editing advancements to enable the development of novel transformative medicines for patients in need. It is important to note that we have a large portfolio of foundational U. S. And international patents covering CRISPRCas9 in human therapeutics, Only some of which are subject to interference proceedings, and we are confident that our IP portfolio will provide meaningful value in the future. We are the exclusive licensee of Harvard University's and Broad Institute's Cas9 patent estates and Editas is uniquely positioned to issue therapeutic applications.

Speaker 3

Our unique position as the exclusive licensee of this patent estate ensures that we are the party responsible for any licensing discussions as CRISPR Cas9 products enter the market, which given the size of the U. S. Patient market, the number of companies vying to develop CRISPR Cas9 medicines is a substantial position. With our newly sharpened strategic focus, our world class scientists and employees and our keen attention to execution, We continue to build upon the momentum from our clinical readout milestones during the Q4 of 2022. We look forward to updating you on our progress And on the execution of our new strategy throughout the year.

Speaker 3

Now I will turn the call over to Baisong, our Chief Medical Officer. Baisong?

Speaker 4

Thank you, Gilmore. Good morning, everyone. Let's start with 8,301, Ruby study for severe sickle cell disease. As Gilmore mentioned, we continue to enroll and dose patients in the RUBI study. We have activated 20 study sites And enrolled 19 patients, almost double the number of patients enrolled from 3 months ago.

Speaker 4

As we previously shared, we began We are on track to provide an update on the Rouvy clinical data both next month year end, As well as to dose 20 total patients by year end. Turning to clinical data, I'm excited that we will present the Rubi clinical data as an oral presentation at the European Hematology Association or EHA Congress and at our company sponsored webinar in June. The data set were including safety and efficacy data for multiple patients, including 10 months data from the 1st patient treated and 6 months data from the 2nd patient treated, including total hemoglobin fetal hemoglobin. We will also share data on safety, neutrophil and platelet engraftment and vessel 2,500,000,000 VOE from the first four patients. As a reminder, last December, we present initial data from the first two patients treated in the RUBY trial.

Speaker 4

The first patient who had 5 months of follow-up after treatment with EDIT-three zero one showed clinically significant improvements Across all hematological parameters and no BOEs. Specifically, the patient had an increase of fetal hemoglobin fraction To 45.4 percent 5 months after N301 infusion. And the correction of anemia These initial clinical data indicated that ED-three zero one provides patients with high and sustained level of fetal hemoglobin And normal level of total hemoglobin. This clinical observation is consistent with preclinical data, which have demonstrated that targeting of gamma globin promoter enables increases of fetal hemoglobin independent of erythropoietic stress. Given the unique gene editing approach and the mechanism of Action by ED-three zero one.

Speaker 4

Supported by preclinical data and initial clinical data, we continue to believe that ED-three zero one can potentially provide robust clinical benefit to patients with severe sickle cell disease and potentially provide clinical differentiation in the long term. As a reminder, a sustained normal total hemoglobin level It's an important clinical outcome for patients as the correction of anemia can significantly improve quality of life And ameliorate and organ damage. We believe sustained normal level of total hemoglobin Could be a potential point of differentiation for 8,301. Turning to 8,301, edicel Phase onetwo trial For transfusion dependent galactosemia, as Guillermo mentioned earlier, we dosed our first patient in Q1 And the patient has successful neutrophil and plantar engraftment. We remain on track to provide initial clinical As we have done for the RUBI study, we are also taking multiple measures To accelerate the development of ED3014DTP and have strong positive momentum, we have enrolled multiple patients Who have completed a freezes and have their CD34 positive sales edited or are in the process of freezes.

Speaker 4

Recently, I have been traveling around the country visiting our RWBY and Elidel clinical trial sites. I very much appreciate the enthusiasm and the support from the investigators and study sites. I'm pleased with the momentum of 8,301 patient recruitment, apheresis, editing and dosing in both studies. I'm excited to hear from investigators The patient dosed with 8,301 have already seen positive changes in their lives. We look forward to sharing additional updates as the year progresses, including Rubi study data next month and at year end and sharing initial clinical data from EDITEL study by year end.

Speaker 4

Now I will turn the call over to Michelle, our Chief Financial Officer

Speaker 2

Thank you, Beis Ham, and good morning, everyone. I'd like to refer you to our press release issued earlier today for a summary of our financial results Q1 of 2023. I'll take this opportunity to briefly review a few items. Our cash, cash equivalents and marketable securities as of March 31st were $402,000,000 compared to $437,000,000 as of December 31, 2022. We expect our existing cash, cash equivalents and marketable Securities to fund our operating expenses and capital expenditures into 2025.

Speaker 2

Revenue for the Q1 of 2023 was $9,900,000 compared $6,800,000 for the same period last year. The increase is related to the previously announced sale of our oncology assets to Shoreline Biophrances And related licenses, which was completed in January 2023. G and A expenses for the quarter was $23,000,000 $19,500,000 for the Q1 of 2022. The $3,500,000 increase is primarily attributable to increased professional services expenses Support business development activities, partially offset by a decrease in stock compensation expense. R and D expenses this quarter were $38,000,000 which is Flat compared to the Q1 of 2022.

Speaker 2

This reflects a decrease in expenses following the strategic reprioritization of our portfolio, Offset by increased investments to accelerate the development of EDIT-three zero one. This reallocation of capital is in line with our strategic priorities. Overall, Editas remains in strong financial position and our shopping discovery focus allowed us to concentrate our talent and extend our cash runway into 2025, We provide ample resources to support our continued progress in both of our EDIT-three zero one trials as well as advancing our research efforts

Speaker 3

It has been almost 1 year since I joined Editas. In this time, the company has demonstrated 2 clinical proof of concepts, Including a proof of concept for EDIT-three zero one, which has the potential to be a competitive and differentiated product for the treatment of sickle cell disease and transfusion dependent beta thalassemia. In addition, as I stated in my opening remarks, we've taken a number of tangible steps to reshape the company around our new strategy, which we shared in early January and have begun executing on that strategy. And this is just the beginning. We look forward to continuing our transformation on sharing our progress with you.

Speaker 3

As a reminder, our strategic objectives for the year include providing clinical updates from the EDIT-three zero one Ruby study in June and end of 2023, Providing clinical data from EDIT-three zero one EDITAL trial for TDT by the end of 2023, dosing 20 total patients in our EDIT-three zero one RUBI study by year end, Hiring a new CSO with specific expertise aligned to our vision, advancing discovery of in vivo editing of hematopoietic stem cells and other tissues, And finally, leveraging our robust IP portfolio and business development activities to drive value and complement our gene editing technology capabilities. I thank all the patients, investigators and our employees who are helping to drive our strategy forward. Thank you very much for your interest in Editas and we're happy to answer questions. Thank you.

Operator

Thank you. At this time, we will be conducting a question and answer session. And our first question comes from Joon Lee with Truist Securities. Please proceed with your question.

Speaker 5

Hi. Thanks for taking our questions. Novartis recently terminated their sickle cell disease program after treating a couple of patients who showed no benefit. Given your editing strategy is similar to what Novartis did, can you point to some differences why your promoter editing strategies should continue to work And I have a follow-up. Thank you.

Speaker 3

Thanks very much, June. Yes, we did actually see that event some weeks or months ago. I think there are some elements that are critical. I think the first thing To point out that it is that in our initial POC readout at the end of last year, we actually saw very robust Data with increase in total hemoglobin early increase in total hemoglobin as well as a robust fetal hemoglobin expression Completely consistent with the preclinical data that were generated in testing our and now our clinical hypotheses that our unique approach of combining an AS Cas12a effector CRISPR enzyme With the targeting of a different region of the HBG-one, two promoter, Which was much closer, in fact, actually encompasses the area in which we see deletions or mutations associated with hereditary persistence fetal hemoglobin. We believe that all those factors, point towards a key difference and differentiation.

Speaker 3

And indeed, Our preclinical data and our clinical data have actually supported that hypothesis.

Speaker 5

Great. So Can you remind me if you have any data preclinical, having comparing the editing with The same region using CAST-nine versus ASC-twelve that you're using and what the differences in outlook maybe?

Speaker 4

Sorry, to interrupt.

Speaker 5

Yes. So I mean comparing Cas9, which is what I think Novartis used And ASCAS12A, which is what you're using, do you get a different outcome if you were to target the same region In terms of getting deletions or inbels?

Speaker 4

Yes. Jun, I think you had a little breakdown, but let me try this is a bit song. Let me try to see I understand correctly. So I think your question to say compared to Novartis and do you have a comparison between F9 and case 12A and also the region just Gilmore mentioned. Is that your question?

Speaker 5

Yes. No, actually, if you target the same region either with CAS9 Or cash 12, Amy? What differences in outcomes do you get?

Speaker 4

Yes. Let me just comment in it We did a comparison. We did from clinical perspective, we scanned a large region Over the promoter region to identify which area to do the editing. So without too many specifics, but We cover the large region of the promoter in the SBG-one hundred and ninety two. And we find out the to the vision we selected, which Gilmore just mentioned, is consistent to the clinical observation for These HBFH.

Speaker 4

And then we also compare the Cas9 with Cas12 We find the difference between Cas9 and Cas12. Does that answer your question?

Speaker 5

Yes. No, absolutely, your Impaired data is fine. That's it. But just curious what was driving that difference. Thank you so much.

Speaker 5

I'll hop back on the queue.

Speaker 4

Thank you.

Operator

Thank you. Our next question is from Samantha Simankow with Citi. Please proceed with your question.

Speaker 6

Hi, good morning and thanks for taking the question. Just a couple for me. For the presentation at EHA, is that a late breaking presentation? I just wanted to clarify.

Speaker 3

Thanks, Samantha. I'll just have based on Update, you'll give you detail there.

Speaker 4

It is a normal oral presentation that being accepted.

Speaker 6

Okay, got it. Thank you for that. And then I also wanted to clarify, I heard you mention, obviously, we'll have updated data for the 1st and second patients. And then I heard you, I think, 4 patients, so will it be 6 patients total that will get data on VOE or is it or VOCs or is it 4 patients total?

Speaker 4

Total will be 4 patients at the Yeehaw presentation.

Speaker 6

Got it. Okay. And then when you're making that cut off for those It was the cut off there. So I'm just curious, is it a couple of months or is it 1 month? Any information you could provide would be helpful.

Speaker 4

Yes. We are for this next two patients, we have 2 months or more.

Speaker 6

Perfect. Thank you so much for taking the questions.

Speaker 7

Thank you. Just to follow-up one

Speaker 3

other thing, I think it's important to understand that obviously we will because the abstract, Which will be published later, was based on data cut earlier. We will actually be presenting more data than in the abstract At the EHA Congress.

Speaker 4

Yes. Thanks, Gilmore. And just kind of also follow-up on that. The app sec will be available on May 11.

Operator

Thank you. Next question is from Dae Gon with Stifel. Please proceed.

Speaker 8

Hey, great. Thanks So much for taking the question and look forward to the data update next month. So I guess I was just kind of Wondering about your strategy going forward. So maybe if you can kind of walk us through how you're thinking about next programs or priorities beyond at a301. I Gilmore, you mentioned in vivo HSC editing.

Speaker 8

But curious, is delivery tech or less burdensome conditioning a stronger emphasis in your lineup? Or is it advancing new programs? And if it's the latter, I guess, would you And if it's the latter, I guess, would you continue to do other ex vivo HSC or is it more of an in vivo? And in that case, would you also think about other organs? And I've got a follow-up.

Speaker 3

Yes. Thanks very much, Jai Gon. We are the large our discovery focus is actually on in vivo. I think that was a very important part and pivot of our strategy Because we believe that it maximizes the or maximizes our ability to exploit the powerful technology that we have available to us. From a point of HSCs, if you reduce the problem of in vivo to sort of 3 elements, Selecting a robust effector molecule or enzyme, CRISPR enzyme, selecting a good target And then delivery, we believe that we have solved 2 of those problems, with very robust human data in the use of our 12a, CRISPR enzyme and the target for that specific HBG-onefour promoter.

Speaker 3

And so that reduces it to an in vivo delivery problem. As I said in my earlier remarks, Our discovery group is actually working in that and we look forward to updating more at an appropriate time in the future. I will say that we are looking actually also beyond HSCs to other tissues. And again, we'll give further updates in the future.

Speaker 8

Got it. Thanks so much. And then second question, I just wanted to follow-up on Baizang's commentary during prepared remarks. So As you were going into the field and kind of gauging physicians take on EDIT-three zero one, you expressed or you commented on their high enthusiasm. Wondering if you could comment, I guess, what proportion of those docs you visited are also looking to administer CTX001?

Speaker 8

And I guess, has there been any kind of gauge or ascertainment from your part as to what their sort of motivation would be in taking CTX-one, like are they lining up patients right now for CTX-one? What kind of sentiment do you have? Are there any reservations on that approach? Any thoughts on that would

Speaker 7

be helpful. Thanks so much.

Speaker 4

Thanks for that question. Yes, I visited quite a few number of study sites. Actually, Many of them are being participating in the previous gene editing trials. So they are very enthusiastic About the approach we're taking, including the different targeting region for editing And the different enzyme to do. And so actually the benefit for us is those investigators have a lot of experience in this field.

Speaker 3

Yes. I think just building on Beisong's remarks, as you're obviously looking out towards the evolution of the market, against the background of enthusiasm for our investigators and indeed the patients With the increase in our acceleration enrollment, we anticipate that in the future that the vast majority of patients We'll be awaiting dosing, at the time of our launch, and I can go into more details on that. But I think a very important point, I think something that has really resonated with the investigators is that our initial clinical data were very encouraging As presented in December, consistent with our preclinical data, and we're actually very confident that we will see replication In subsequent patients as we continue to monitor them through the execution of the 301 studies.

Speaker 8

Great. Thanks for taking our

Speaker 7

questions.

Operator

Thank you. Our next question comes from Steve Seedhouse with Raymond James. Please proceed with your question.

Speaker 7

Good morning. Thanks so much.

Speaker 9

My question actually requires a bit of a prelude, so I hope everyone can bear it for a moment. You made a comment on globin locus Editing increasing fetal hemoglobin independent of urethra poetics stress. And as you know, the recent ICER report on exocell uncovered An ongoing phlebotomy, at least 1 in sickle cell and some of the earlier data releases for that program in thalassemia indicated phlebotomy use there as well. But that just stopped being reported at some moment. So it's not clear how prevalent phlebotomy use is for exacel.

Speaker 9

And this is all important because There was data at ASH years ago, as I'm sure you also know, indicating BCL11A editing cooperates with phlebotomy in primates To accentuate F cells and ultimately HBF levels, probably because of the stress erythropoiesis that causes. So All that said, I'm curious if you agree that phlebotomy is potentially confounding fetal hemoglobin data for BCL11A approaches. And if you know what is the impact specifically for your editing approach at the HPG-twelve locus, What has phlebotomy used been like in your study and if you think this is all potentially competitive advantage for you? Thanks.

Speaker 4

Steve, thank you very much for your question. So from our own clinical data, preclinical data and also published you're probably referring to the BCL11A targeting approach, it did require some stimulation For the Aries reprioriatic stress to increase the fetal lubine sufficient fetal lubine expression. And so that's actually the reason we're choosing the target that we are treating now and we actually just take a longer to get all the targets from clinical preclinical study perspective and that's being validated by other publications. And regarding specific clinical data for the BCL11 approach, I have not seen formal publication. So I would waiting for them to publish their data and to see where we have a better understanding.

Speaker 4

So I would not comment on their data unless they publish.

Speaker 3

However, it is worth pointing out that in our early disclosure, we actually noticed a combination of very robust normalization or correction of anemia in our first patient in December, and that was associated with a robust fetal hemoglobin expression suggesting that indeed stressorrhythoesis as we hypothesized based on the known biology And our nonclinical data, that our approach is not dependent on stressorrhizae.

Operator

Thank you. Our next question is from Yanan Zhu with Wells Fargo. Please proceed with your question.

Speaker 10

Hi, thanks for taking our questions. Maybe to continue the discussion a little bit from the prior question. Gilmore, you mentioned that the total hemoglobin for the first patient It is quite robust and in the normal range. The percent of fetal hemoglobin Appears to be very much in line with competitor gene editing product. So I was wondering is the greater Total hemoglobin reported for that patient, is that due to the total number of For red blood cells, is that could that potentially be a reason?

Speaker 10

Or could it be related to the baseline level of hemoglobin in that patient. And along that line to continue a little further and to Perhaps looking into what we could expect from patient number 2 at EHA, I was just wondering, could you remind us The baseline total baseline hemoglobin for patient number 2 and what is the normal range For the female patient, which is obviously the second patient. Thank you.

Speaker 4

Thanks. So thanks for your question. And then so I will start We've answered your first part of the question regarding the fetal hemoglobin versus the number of red blood cells and all these. So what we see is actually robust erythropoiesis for these patients we observed. So their hemoglobin level It's contributed by both the hemoglobin per cell as well as the number of red blood cells.

Speaker 4

And you can see that we actually do see the increase also on both end of that. And then also I want to mention that even though we also have like around 45% of fetal hemoglobin And because of the total hemoglobin level is high and the total amount of fetal hemoglobin is also high. So that's kind of all in that. And then regarding the second patient, and we will present the data very soon. So I would not comment on the specifics of the patient data, But I can mention that, of course, the male and female normal hemoglobin level are different.

Speaker 4

Usually for male is Around 13.5 to up to 18 gram per deciliter. For female, it's around 12 to 14 and depending on the reference lab. So there's some difference in that too.

Speaker 3

Yes. I think one other thing, and you asked a question about, does what was the baseline hemoglobin of The patient 1, and I think what we can say is that the hemoglobin or the total hemoglobin increase that we saw occur very rapidly just within the 1st few months Of dosing, comprised at 3.5 gram per deciliter increase.

Speaker 4

Yes. Maybe I'll just add a little bit nuance on that baseline for sickle cell patient is basically for gene editing trial. And because the patient when they prepare for apheresis and conditioning, they usually have blood transfusion. So the baseline actually we have is not the lowest level we recorded. We just set up the time of the VASIL 2 as a baseline.

Speaker 4

So actually it's compounded. It could be many different reasons for the baseline on that too. That's just a nuance. It's lower. It's actually it's lower than it's around a little bit over 10 grams per deciliter When we actually on our record for this patient, this is just example about the baseline maybe a little bit more confusing.

Speaker 10

Very nice. Thank you for all the explanation. Maybe a quick follow-up. Do you expect this to be Also a differentiator for PDT and perhaps maybe at a greater level of Because anemia is a major manifestation of that disease. Thank you.

Speaker 3

Thanks very much, Ynon. We designed, our discovery group and scientists designed And selected the combination of Cas12a with the specific targeting of the HbG1-two promoter Using a set of empirical experiments, to determine what was the best approach to driving not just fetal hemoglobin expression, What robust erythroids output that would be or red cell output for the bone marrow that would be independent of stress erythropoiesis or anemia. And those empirical experiments really determined that approaching or Directly targeting the HBG-onefour promoter would be better. And that was the original design hypothesis. The nonclinical data, preclinical data actually supported that, showing robust erythroid output In comparison to other approaches, as well as robust fetal hemoglobin expression.

Speaker 3

And indeed, that is what we have seen, As we disclosed in our first sharing of the data or cut the data from Our RUBY study. And so obviously, it is we haven't seen enough data in our Edithal patient. But What I can say is that Ruby has certainly demonstrated data that are consistent with both the preclinical data, which were supportive Of the original biological and therapeutic hypotheses.

Speaker 10

Great. Thanks for all the answers.

Operator

Thank you. Our next question is from Phil Nadeau with Citi Cowen, please proceed with your question.

Speaker 11

Good morning. This is Ernie Rodriguez for sales. Thanks for taking my question. On the sickle cell program, have you met with the FDA to gain better visibility on the regulatory path? And then second question on the TTP program.

Speaker 11

For the year end update, would that include only the sentinel Patients that you initially dose or will that or will you disclose additional patients? And if you will be disclosing additional patients, Are you planning on reporting when you switch from sentinel dosing to viral dosing? Thank you.

Speaker 3

So, I think what I'll do is ask Baisong to I've actually I'll keep track of the questions, Baisong. If I'd ask you just to Yes. So the question around the FDA and the regulatory interactions that are planned.

Speaker 4

Yes. Thanks for your question. So We certainly have a lot of engagement with FDA. As you see that recently, we have the orphan drug designation. And from the registration perspective, we previously announced that we actually have the alignment on the potency assay with FDA which will FDA will consider this efficacy that data can be supporting registration.

Speaker 4

And we will have further engagement with the agency To align on the total registration package for the BLA submission, which is also planned. And your second part of the question is about the beta cell data. So we are Moving really along with the EDITEL study, we expect that we will have data by year end More than Sentinel patients. And so we're looking forward to sharing this data by the year end.

Speaker 11

All right. I see. And you are more than a second question. So are you planning to Disclose when you get approved to continue parallel dosing before then?

Speaker 3

Okay. So, what we are actually planning to do is I think the key thing is we're on track To get the data for readout at the end of the year for that initial readout at the end of the year, we haven't determined if We're actually going to share that. But I think the important point is that we are well on track to Disclose good initial data for the end of the year.

Speaker 4

Got it. Thank you.

Operator

Thank you. Our next question comes from Rick Bankowski with Cantor Fitzgerald, please proceed with your question.

Speaker 12

Hey, good morning and congrats on all the progress. I guess I'll expand a bit on the last question on the test towards registration for EDIT-three zero one. 20 patients is a pretty substantial cohort size in sickle cell disease. So do you have any sense of how many patients worth of data you will need for a registrational filing?

Speaker 4

Thank you for the question. So we certainly think that with the gene editing approach that We have we will be able to generate substantial amount of data. And the specifics on The number of patients to be able to use for such registration, we need to align with the regulatory agencies. So we are planning to discuss with FDA.

Speaker 12

Okay, got it. And I just have another quick one. I was hoping for a little bit more granularity on the Collaborative revenues for the quarter, were all of the $9,900,000 in revenue attributable to the Shoreline transaction or are there other revenues attributable to other partnerships in there?

Speaker 2

It's a combination of both the Shoreline and then some other Small, like sublicense revenue.

Speaker 12

Okay, got it. Thanks for taking the questions.

Speaker 3

Thank you.

Operator

Thank you. Our next question is from Rich Law with Credit Suisse.

Speaker 7

I have a couple of questions for you guys. So with the Appeal litigation pending, what does it mean for companies such as CRISPR and Vertex that already filed a BLA for XSL that utilize CRISPR Cas9 from your IP perspective? They don't have a license from you or the Bro and could potentially launch the product before we know the outcome of the appeal. So any insight here would be helpful. And then I have a follow-up question.

Speaker 3

Okay. Thanks very much, Rich. I think the key thing is that we sort of anticipate the judgment in the early to mid-twenty 24. We are confident that we prevail as we have before, Largely because what are under discussion is the application of the law and not about new facts And it's the application of the law by PTAB. Now setting aside that interference, I think the important thing is to say that we have a portfolio of IP Not subject to any interference.

Speaker 3

That actually covers products in development using Cas9 For the application of human therapeutics and looking forward, we are happy to grant licenses to enable delivery of this technology to patients And believe that we should recognize significant value around that.

Speaker 7

Okay, great. Thanks. So in terms of granting license, so we're not going to know the appeal decision Likely before the BLA and also potentially the launch. Like how do you sort of think about that?

Speaker 3

Well, I think that there are a number of important points to make. I think the first again is just to remind that the appeal applies to some Of our Cas9 in human therapeutics IP estate, but not all. I think it's important To emphasize that we have Cas9 or IP around Cas9 use in human therapeutics That is not subject to any interference and therefore is not subject to that appeals case. And we actually believe that it actually covers products in development. And so What I think I want people to really understand is that, that appeals case is around interference on some of our IPOs statement, not all.

Speaker 7

Okay. Got it. And then just one more question from me. So you're seeing some next gen CD therapies already in development with new conditioning agents. So it don't succeed.

Speaker 7

It doesn't seem like the shelf life for these 1st gen therapies will last too long. Any thoughts about this?

Speaker 3

So just to be clear, I understand your question. You're actually questioning if The evolution of new conditioning would actually change the landscape for the products that are either Are close to approval. I think it really very much depends on the nature of the conditioning. As we look out at toroxicity, obviously, something we've looked at closely. One of the important things is, to balance Both the reduction of toxicities with engraftment efficacy, and I think we all see that it's a very important path To increasing eligibility for patients because more patients will be able to tolerate a non genotoxic, less Toxic conditioning regime.

Speaker 3

Many of the regimes or some of the approaches are not actually editing dependent. They are actually And so what we actually believe is that, the evolution of milder conditioning could actually expand And grow the size of the eligible patient population for all. I think the important thing obviously also is that We are looking beyond not just conditioning, but looking to in vivo editing as part of our strategy. For the very simple reason that we believe that in vivo editing would further increase the eligibility of the patient population for treatment.

Speaker 7

Got it. Very helpful. Thanks.

Operator

Thank you. Our next question is from Debjit Chattopadhyay with Guggenheim. Please proceed with your question.

Speaker 13

Good morning, everyone. This is Ry Forsyth on for Debjit. I just want to build off of the conditioning discussion and sort of Get an outline of your strategy. Is it sort of bifurcated, kind of exploring both in vivo editing and the opportunity In license, assets that would be alternative to busulfan. Can you sort of map that out for us?

Speaker 13

And just wanted to get your thoughts to, on the ASGCT GCP abstracts, and sort of what you see in the competitive landscape around conditioning, especially given that a competitor is Kind of moving forward with the CD117 approach.

Speaker 3

Yes. Thanks very much, Debjit. I think I'll start and then I will have a base on comment. From a strategic point of view, we are using a 2 pronged strategy. We have Directed investment, significant investment internally to our discovery of in vivo editing for Hematopoietic stem cells.

Speaker 3

And I think as I said earlier, we believe that this is a problem That we can focus on where we can focus on delivery, where certainly in humans, we believe we have solved the 2 of the 3 challenges around the selection of a CRISPR enzyme as well as a target. In parallel, we actually are Continuing have ongoing evaluations of milder conditioning approaches. And I think you asked more importantly Or in follow-up, a question about the, for example, CD-one hundred and seventeen, I'll ask Baesung just to talk about that.

Speaker 4

Yes. Thanks for your question. I can say that we have looked into these smart condition in very deep, Looking this space as well as internal efforts wise. And there were generally probably 2 approaches. One is that CDWonman 7 antibody and in that direction and the other one is actually Doing the cell modification together with the gene editing.

Speaker 4

So that approach is still in infancy, if I may say. And the previous approach with antibody have many different exercise on that. So I think we are very closely monitoring this space and understand this. And I also want to mention that the milder conditioning, If successful is not going to be only successful for sickle cell transplant, it's going to be successful for leukemia and many different gene the So, pretty good area. So, we are actually very much looking into this space.

Speaker 13

Thanks for the insight.

Operator

Thank you. Our next question is from Madhu Kumar with Goldman Sachs. Please proceed with your question.

Speaker 14

Hey, this is Rob on for Madhu. Thanks for taking our question. We were just wondering how should we think about forward OpEx, given forward OpEx, particularly R and D, given our robust

Speaker 3

I'm sorry, Rob. I actually had great difficulty hearing your question. Could you just repeat it, please?

Speaker 14

Sure. We are just wondering, how we should be thinking about forward OpEx, particularly spending In regard to transfusions versus follow-up?

Speaker 3

Okay. So I think you're asking about forward looking OpEx around the execution of the RWBY study. Is that correct? Okay. Michelle?

Speaker 2

Yes. So Rob, I mean, we don't disclose our annual OpEx or our quarterly OpEx, but I can tell you that about half of our spend is in on the both the RUBY trial and the TDC trial. So We don't expect an enormous increase quarter over quarter. But as we As we do dose more patients, obviously, our EV spend will go up, but not substantially.

Speaker 4

Thank you.

Speaker 2

And our current, I'll just say one more, our current cash runway Support, moving child.

Operator

Thank you. Our next question is from Greg Harrison with Bank of America, please proceed with your

Speaker 6

Good morning. This is Mary Kate on for Greg. Thanks for taking our question. So with 19 patients enrolled and plans for 20 to be dosed by year end, maybe how many sickle cell patients have been Currently treated with EDIT-three zero one and maybe how could we expect to see this represented in the efficacy readout by the year end update? Thank you.

Speaker 4

Thanks for the question, Mary. So we have 19 patients enrolled and among those, As we just mentioned, 4 have been dosed and we actually have more patients have been completely freezes, have a CD34 Sales edited and ready to schedule dosing. And then we have other patients who are in the process of freezes. And so we are very Confident that we will be able to dose 20 patients by the end.

Operator

Great. Thank you. Thank you. Our next question is from Luca Giuseppe with RBC Capital Markets. Please proceed with your question.

Speaker 15

Great. Thanks so much for taking my questions. Maybe on bisthalassemia, obviously, most patients are in Southern Europe. Wondering if you could comment on what's the plan to capture that market and maybe how you're thinking about some of the key lesson learned from the unsuccessful launch And then maybe on sickle cell disease, wondering if you can comment on pricing. Obviously, the active reports suggest $1,900,000 So wondering if that is actually aligned And then maybe lastly on LCA10, any update on partner discussions there?

Speaker 15

Thanks so much.

Speaker 3

Thanks very much, Luca. So obviously beta thalassemia is a disease that dominates parts of the world, particularly Europe, Southeast Asia, South Asia, amongst others. From a point of view of our forward looking, We are actually focusing our efforts on North America currently. We have shared in the past that from an upside point of view, We are looking open to partnering, ideally targeting a partner with a large global footprint Who would actually collaborate certainly in sort of ex U. S.

Speaker 3

Development and commercialization. So that's What I would say, I can talk about, when we look to your point about beta thalassemia outside North America, I will say that we are happy, Very happy with the progress that we're making with execution here within the United States and North America. With regard to pricing, I think It's very early days yet for us to be talking about pricing. This is something that we would be very happy to discuss when we're actually closer to approval and launching and we look forward to future conversation around there. Obviously, we look to the market evolution over that time, but We're going to talk about that closer to launch and approval.

Speaker 3

And then finally, with regard to LCA10, We have we really have a practice of not really going into details until we have a deal Signed and executed.

Speaker 4

Maybe just to

Speaker 11

add one more

Speaker 4

point.

Speaker 15

Please.

Speaker 4

As Gilmore mentioned about pricing, right? So certainly we're very early stage and we're happy to see the community is looking to the value of this gene editing therapy And we are happy to see that the ICER report recently in this space. So we as Gilmore mentioned, this will be evolving, but we are pleased to see that The entire community recognizes the value of the medicine in this field. Thank you.

Speaker 3

Thanks so much.

Operator

Thank you. Our next question is from Jay Olson with Oppenheimer. Please proceed with your question.

Speaker 5

Hey, good morning. This is Chung on the line for Jay. Thanks for taking the question. Maybe 2 from us. So I'm just wondering if There's a chance where you have a capacity to dose more than 20 patients for the sickle cell disease program this year.

Speaker 5

And second question is on your ex U. S. Strategy. What's your current thinking? And if you are Planning to partner that program, what is the best timing to do that?

Speaker 5

Thank you.

Speaker 3

Thanks very much. So do we have capacity to dose more than 20 patients? Yes. One of the important points when we Rode out our strategy was to again sharpen our focus on developing and Accelerating 301 and indeed we have deployed capital to enable not just the acceleration on the clinical Saiid, but actually also to ensure that we have capacity to edit or other CMC capacity to edit and support those that clinical acceleration. So indeed, we do have that capacity To dose more than 20 patients.

Speaker 3

And then I think your second question was around ex U. S. And the timing partnership. I think as I said before, we are interested in partnering. We're looking to a partner with a global footprint that would actually certainly support ex U.

Speaker 3

S, particularly on the development and commercialization. And with regard to timing, we wouldn't really discuss the timing until we actually have a deal signed and executed.

Speaker 5

Okay. Thanks.

Operator

Thank you. Our next question is from Joel Beatty with Baird. Please proceed with your question.

Speaker 13

Hello. This is Benjamin Paluch on for Joel. Thanks for taking our question. Looking across other late stage In sickle cell and TdT, it appears that data sets of 30 patients could support approval. So with being on track to dose 20 patients by year end, how quickly do you think you may able to secure the necessary data to support regulatory approvals?

Speaker 13

Thank you.

Speaker 3

Thanks very much, Ben. Fei Song?

Speaker 4

Yes. Thanks for the question. As I mentioned earlier, in terms of total number of required to support registration and then need to have a required alignment with the regulatory agency. And in terms of the progression of the Study, we are very positive about the momentum. So we are very optimistic.

Speaker 4

We will be able to dose patients as we planned.

Speaker 3

So I think the key thing Ben is that you've actually identified sort of a benchmark. But obviously, what we need to do is, as planned, sit with the regulators and

Speaker 5

Great. Thank you.

Operator

Thank you. Our next question is from Joon Lee with Truist Securities. Please proceed with your question.

Speaker 5

Hey, thanks for taking the follow-up question and sorry for the background noise about the translation. So I had the same question as Steve, but maybe a different way of asking. What percentage of Stifel patients with hereditary persistence of fetal hemoglobin have mutations along the globin locus versus the BCL11A

Speaker 4

Jim, thank you for the question. So We do not have all the specifics on your question on that, but we know is the mutation in the Promoted region directly impacted the fetal globin expression. But BCLA is a transcription factor, Which impacted multiple different cell limits and that the mutation of the BCG11a will have much different impact From the fetal globin and expression only will have other impacts too. So the gene editing mutation is much more complicated issue And then the HBFH with the promoter region and mutation for the gamma globin promoters.

Speaker 3

I think another way, June, to actually characterize this Because some of the prevalence is a little harder to quantify, but another way of pointing it is really the strength of the signal. The hereditary persistence of fetal hemoglobin and its capacity to substantially mitigate the effects of sickle cell disease and thalassemia were actually determined actually Quite some time ago because this phenotypic change, the phenotype to genotype correlation It was actually quite robust and identified actually a few decades ago, whereas the BC11a was identified really through a GWAS Assessment, genome wide assessment.

Speaker 5

Thank you.

Operator

Thank you. We have reached the end of the Q and A session. And with that, ladies and gentlemen, this concludes today's call. Thank you once again for your participation. You may now disconnect your lines.

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Earnings Conference Call
Editas Medicine Q1 2023
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